The SPACE clinical trial took place between 2016 and 2017 in the Netherlands. This single-centre, phase II, cross-over trial evaluated an 8-week course of pyridostigmine versus placebo in 35 people with SMA types II to IV.
The results, published in December 2022, show:
- no statistically significant difference in Nine-hole peg test (NHPT) and motor function measurement (MFM) results with pyridostigmine and placebo;
- good tolerance of pyridostigmine;
- a majority of participants (74.4%) reported a moderate or significant benefit of pyridostigmine on their fatigability (versus 29.7% on placebo).